How comparable are patient outcomes in the &quot;real-world&quot; with populations studied in pivotal AML trials?

Tiong, IS; Wall, M; Bajel, A; Kalro, A; Fleming, S; Roberts, AW; Thiagarajah, N; Chua, CC; Latimer, M; Yeung, D; Marlton, P; Johnston, A; Enjeti, A; Fong, CY; Cull, G; Larsen, S; Kennedy, G; Schwarer, A; Kipp, D; Ramanathan, S; Verner, E; Tiley, C; Morris, E; Hahn, U; Moore, J; Taper, J; Purtill, D; Warburton, P; Stevenson, W; Murphy, N; Tan, P; Beligaswatte, A; Mutsando, H; Hertzberg, M; Shortt, J; Szabo, F; Dunne, K; Wei, AH

How comparable are patient outcomes in the "real-world" with populations studied in pivotal AML trials?

Author(s): Tiong, IS; Wall, M; Bajel, A; Kalro, A; Fleming, S; Roberts, AW; Thiagarajah, N; Chua, CC; Latimer, M; Yeung, D; Marlton, P; Johnston, A; Enjeti, A; Fong, CY; Cull, G; Larsen, S; Kennedy, G; Schwarer, A; Kipp, D; Ramanathan, S; Verner, E; Tiley, C; Morris, E; Hahn, U; Moore, J; Taper, J; Purtill, D; Warburton, P; Stevenson, W; Murphy, N; Tan, P; Beligaswatte, A; Mutsando, H; Hertzberg, M; Shortt, J; Szabo, F; Dunne, K; Wei, AH;
Details: Publication Year 2024-03-26,Volume 14,Issue #1,Page 54
Journal Title: Blood Cancer Journal
Publication Type: Mar 26 epub ahead of print
Abstract: Despite an increasing desire to use historical cohorts as "synthetic" controls for new drug evaluation, limited data exist regarding the comparability of real-world outcomes to those in clinical trials. Governmental cancer data often lacks details on treatment, response, and molecular characterization of disease sub-groups. The Australasian Leukaemia and Lymphoma Group National Blood Cancer Registry (ALLG NBCR) includes source information on morphology, cytogenetics, flow cytometry, and molecular features linked to treatment received (including transplantation), response to treatment, relapse, and survival outcome. Using data from 942 AML patients enrolled between 2012-2018, we assessed age and disease-matched control and interventional populations from published randomized trials that led to the registration of midostaurin, gemtuzumab ozogamicin, CPX-351, oral azacitidine, and venetoclax. Our analyses highlight important differences in real-world outcomes compared to clinical trial populations, including variations in anthracycline type, cytarabine intensity and scheduling during consolidation, and the frequency of allogeneic hematopoietic cell transplantation in first remission. Although real-world outcomes were comparable to some published studies, notable differences were apparent in others. If historical datasets were used to assess the impact of novel therapies, this work underscores the need to assess diverse datasets to enable geographic differences in treatment outcomes to be accounted for.
Publisher: NPG
Keywords: Humans; Neoplasm Recurrence, Local/drug therapy; Treatment Outcome; Cytarabine/therapeutic use; Gemtuzumab/therapeutic use; *Hematopoietic Stem Cell Transplantation; *Leukemia, Myeloid, Acute/therapy; Antineoplastic Combined Chemotherapy Protocols/therapeutic use
Research Division(s): Blood Cells And Blood Cancer
PubMed ID: 38531863
Publisher's Version: https://doi.org/10.1038/s41408-024-00996-x
Open Access at Publisher's Site: https://doi.org/10.1038/s41408-024-00996-x
Terms of Use/Rights Notice: Refer to copyright notice on published article.

Creation Date: 2024-04-08 12:14:56

Last Modified: 2024-04-08 02:53:53